Navigation Links
Bascom Palmer Eye Institute announces breakthrough for degenerative vision disorder

Miami - A research team, led by John Guy, M.D., professor of ophthalmology at Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, has pioneered a novel technological treatment for Leber Hereditary Optic Neuropathy (LHON), an inherited genetic defect that causes rapid, permanent, and bilateral loss of vision in people of all ages, but primarily males ages 20-40. Genetic mutations in the mitochondria (part of the cell that produces energy) cause the disorder. Currently, there is no cure for LHON. However, Guy and his team have successfully modified a virus and used it to introduce healthy genes into the mitochondria to correct the genetic defect. Using experimental models, they have proven that it is both safe and effective to replace mutated genes with healthy ones and that doing so prevents deterioration of the retinal cells that form the optic nerve. This research demonstrates that when efficiently introduced into mitochondria, normal DNA can correct a biochemical defect in cellular energy production and restore visual function.

"A wide range of other factors, including aging, cancer, and Parkinson's disease, are also caused by mutations in the mitochondria," said Dr. Guy. "This new approach shows the vast potential for genetic-therapy applications, while helping to address a significant cause of blindness."

The healthy genes were delivered into the mitochondria via an innovative viral delivery system. Specifically, Guy redirected the adeno-associated virus (a small virus that infects humans but is not known to cause disease) to the mitochondria rather than to its typical target, the nucleus, where most genes are housed within the cell. He did so via a mitochondrial-targeting sequence (a peptide chain that directs the transport of a protein). This permitted the replacement of the defective mitochondrial gene with a healthy one, which then restored energy production to the affected ocular cells. Two National Institutes of Health/National Eye Institute grants, totaling $6.1 million funded this research, which began in 2007.

"Other research studies have shown that LHON patients who have lost their vision still have some sensitivity to light," said Guy. "This indicated that if you can restore the functioning of those cells through gene therapy, those patients could see again." In conjunction with his research, Guy explored why only about 50 percent of patients with the genetic mutation develop LHON, while others do not.

Known for exploring gene therapy as a potential treatment for diseases of the optic nerve, Guy holds several patents related to mitochondrial gene therapy biotechnology. His next steps will be to investigate incorporating all three genes that cause LHON into a single viral carrier and hopefully receive FDA approval to inject therapeutic genes into patients who have visual loss from mitochondrial disease.

On April 20, 2012, Proceedings of the National Academy of Sciences of the United States of America (PNAS) - one of the world's most-cited multidisciplinary scientific serials - published an article by Guy about this recent breakthrough. Click here to read the article.

Contact: Ann Carney
Bascom Palmer Eye Institute

Related medicine news :

1. The Arnold Palmer Invitational Presented by MasterCard Expands Relationship with MasterCard to Offer Exclusive Prepaid Business Gift Cards
2. Medical Training School, Pima Medical Institute Offering First Bachelor's Degree Program
3. Dana-Farber and Sanford-Burnham Institute license flu-targeting antibodies to Genentech and Roche
4. Laser Spine Institute Honored at the Gridiron Greats Night of Champions
5. Akron Institute of Herzing University Launches Its First Bachelors Degree Programs to Prepare Students for Even Greater Success in Business, Health Care and IT
6. Carpet and Rug Institute Expands Seal of Approval Program
7. San Diego State University and BIOCOM Institute Receive $4.95 Million Grant: The BRIDGE Project, Linking Education to Employment in San Diegos Life Sciences Industry
8. Northern California Cancer Center Changes Name to Cancer Prevention Institute of California
9. ChildCare Education Institute Supports the First Ladys Efforts to Reduce Childhood Obesity
10. GREENGUARD Environmental Institute Revamps Web Site
11. JBG to Develop Major Research Campus for National Cancer Institute
Post Your Comments:
(Date:6/24/2016)... ... June 24, 2016 , ... Dr. Amanda Cheng, ... treatment. Dr. Cheng has extensive experience with all areas of orthodontics, including robotic ... osteogenic orthodontics. , Micro-osteoperforation is a revolutionary adjunct to orthodontic treatment. It ...
(Date:6/24/2016)... NY (PRWEB) , ... June 24, 2016 , ... Topical BioMedics, Inc, makers of Topricin ... companies that call for a minimum wage raise to $12 an hour by 2020 and ... This will restore the lost value of the minimum wage, assure the wage floor does ...
(Date:6/24/2016)... ... June 24, 2016 , ... EB Medicine presented ... in Emergency Medicine conference in Ponte Vedra Beach, FL. The awards honor the ... Emergency Medicine Practice and Pediatric Emergency Medicine Practice. , “With this award, ...
(Date:6/24/2016)... Vegas, Nevada (PRWEB) , ... June 24, 2016 ... ... Las Vegas client, The Grove Investment Group (TGIG), has initiated cultivation and processing ... Grove, in Las Vegas and Pahrump, Nevada. , Puradigm is the manufacturer of ...
(Date:6/24/2016)... ... 24, 2016 , ... National recruitment firm Slone Partners is pleased ... and genomics experience, as Vice President of North American Capital Sales at HTG ... leading the sales team in the commercialization of the HTG EdgeSeq system and associated ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... , June 24, 2016 ... the "Structural Electronics 2015-2025: Applications, Technologies, Forecasts" ... In-Mold Electronics, Smart Skin, Structural ... Structural electronics involves electronic and/or ... protective structures, replacing dumb structures such as vehicle ...
(Date:6/24/2016)... India , June 24, 2016 ... Needles Market by Type (Standard Pen Needles, Safety Pen ... Therapy (Insulin, GLP-1, Growth Hormone), Mode of Purchase (Retail, ... by MarketsandMarkets, This report studies the market for the ... expected to reach USD 2.81 Billion by 2021 from ...
(Date:6/23/2016)... 23, 2016 Research and Markets has ... - Forecast to 2022" report to their offering. ... for the patients with kidney failure, it replaces the function ... the patient,s blood and thus the treatment helps to keep ... in balance. Increasing number of ESRD patients ...
Breaking Medicine Technology: